Researchers achieve germline transmission of ‘gene knockdown’ in mice

RNA interference (RNAi) has emerged as an extremely versatile and powerful tool in biomedical research. A new study published in the February issue of Nature Structural Biology reports the creation of transgenic mice in which inherited RNAi lowers or silences the expression of a target gene, producing a stable “gene knockdown.” This finding extends the power of RNAi to genetic studies in live animals, and has far-reaching implications for the study and treatment of many human diseases.

Designer molecules correct RNA splicing defects

With a high-tech fix for faulty cellular editing, scientists at Cold Spring Harbor Laboratory have moved a step closer to developing treatments for a host of diseases as diverse as breast cancer, muscular dystrophy, and cystic fibrosis. Many human diseases have been linked to defects in a cellular editing process called pre-messenger RNA splicing. Adrian Krainer, a molecular biologist at Cold Spring Harbor Laboratory, has spent years investigating this complex editing process, which takes the information coded in genes and makes it available for building proteins. In a new study published in the journal Nature Structural Biology, Krainer’s team has devised a clever way to correct RNA splicing defects implicated in breast cancer and spinal muscular atrophy (a neurodegenerative disease). In principle, the technique could provide the ability to correct RNA splicing defects associated with any gene or disease.